'SSADHD' Affecting 1 in 1 Million
Meeting to Develop Patient-Customized Medication
Patients, Medical Staff, and FDA Officials Gather
[Asia Economy Reporter Chunhee Lee] GC Green Cross announced on the 31st that it participated as a co-sponsor in the U.S. Food and Drug Administration (FDA)'s 'Externally Led Patient-Focused Drug Development (EL-PFDD)' meeting together with its partner, the U.S. biotech company Speragen, taking time to establish guidance for the development of new drugs for rare and intractable diseases.
EL-PFDD is an expansion of the Patient-Focused Drug Development (PFDD) initiative, which the FDA has been implementing since 2012 to develop treatments for rare diseases, into externally led meetings starting in 2015. These meetings are organized by patient groups and bring together FDA officials, medical professionals, and industry stakeholders to discuss and establish frameworks for benefit-risk assessments in clinical research for treatments.
This EL-PFDD meeting was held regarding the rare intractable disease 'Succinic Semialdehyde Dehydrogenase Deficiency (SSADHD).' SSADHD is a neurodegenerative disease inherited in an autosomal recessive manner due to enzyme deficiency caused by genetic defects. It is known to occur in approximately one in a million people on average, typically manifesting around the age of one. Considering that the World Health Organization (WHO) defines rare diseases as those with a prevalence of less than one in 1,000, SSADHD qualifies as an ultra-rare disease.
A total of 113 stakeholders, including SSADHD patients and their families worldwide, medical professionals, and FDA representatives, attended the meeting. They gathered patient experiences and opinions on the disease and discussed various topics such as which types of treatments could provide the best effects for patients and how to derive effective data for clinical use.
This is the first time a domestic pharmaceutical company has participated in an EL-PFDD meeting. GC Green Cross signed a joint development agreement for an SSADHD treatment with Speragen in July last year and is currently developing a new drug. SSADHD currently has no commercialized treatments, and globally, no development efforts are underway. Patients are only receiving prescriptions for antiepileptic drugs to alleviate seizure symptoms.
The two companies aim to develop the first-in-class treatment for SSADHD and plan to enter clinical trials next year. Since EL-PFDD allows for the collection of opinions from patients, medical professionals, and the FDA, it is expected that the likelihood of success will increase and the development speed will accelerate.
Based on the discussions at this meeting, the SSADHD Patient Association plans to submit a report compiling patients' opinions (Voice of the Patient report) to the FDA by the end of this year. Subsequently, GC Green Cross and Speragen plan to establish clinical outcome assessments for enzyme replacement therapy development for the disease and proceed with clinical trials.
Alice McConnell, CEO of Speragen, expressed gratitude, saying, “We thank the patients and families who courageously shared their experiences living with SSADHD.” Huh Eun-cheol, CEO of GC Green Cross, also stated, “This meeting will be an important milestone toward our goal of providing new treatment options to patients and families suffering from this disease.”
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